NCT05146830
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
Enrolling by Invitation
Not specifiedAges 14–50ObservationalStephanie CherquiInvestigator-initiated
~50 participants
Updated 2024-07-03 on ClinicalTrials.gov
What's tested:Safety and Efficacy Assessments
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured over Baseline to Year 15 post gene therapy
+3 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesCalifornia1
Study leadership
- Stephanie Cherqui, PhD · PRINCIPAL_INVESTIGATOR · University of California, San Diego
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
What this trial measures
- Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs)Baseline to Year 15 post gene therapy
- Number of participants with clinically relevant abnormalities, as assessed by vital sign (heart rate, pulse rate, and temperature)Baseline to Year 15 post gene therapy
- Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests (chemistry and hematology)Baseline to Year 15 post gene therapy
- Number of participants with clinically relevant abnormalities, as assessed by by electrocardiograms (ECGs) (rate, rhythm, intervals)Baseline to Year 15 post gene therapy